Results 31 to 40 of about 89,699 (157)

A New Antisense Phosphoryl Guanidine Oligo-2′-O-Methylribonucleotide Penetrates Into Intracellular Mycobacteria and Suppresses Target Gene Expression

Frontiers in Pharmacology, 2019
The worldwide spread of multidrug-resistant Mycobacterium tuberculosis strains prompted the development of new strategies to combat tuberculosis, one of which is antisense therapy based on targeting bacterial mRNA by oligonucleotide derivatives. However,
Yulia V. Skvortsova, Elena G. Salina, Ekaterina A. Burakova, Ekaterina A. Burakova, Oksana S. Bychenko, Dmitry A. Stetsenko, Dmitry A. Stetsenko, Tatyana L. Azhikina   +7 more
doaj   +1 more source

Antisense Oligonucleotide-Mediated Transcript Knockdown in Zebrafish. [PDF]

PLoS ONE, 2015
Antisense oligonucleotides (ASOs) are synthetic, single-strand RNA-DNA hybrids that induce catalytic degradation of complementary cellular RNAs via RNase H.
Andrea Pauli, Tessa G Montague, Kim A Lennox, Mark A Behlke, Alexander F Schier   +4 more
doaj   +1 more source

Position-Dependent Stabilization of DNA/RNA Duplexes by Site-Specific Incorporation of LNA Nucleosides

Journal of Nucleic Acids
Owing to their enhanced functional properties, 2′,4′-bridged nucleic acids/locked nucleic acids (2′,4′-BNAs/LNAs) are considered promising candidates for antisense oligonucleotide therapeutics.
Elisa Tomita-Sudo, Tomoka Akita, Nae Sakimoto, Saori Tahara-Takamine, Renshin Sano, Shigenori Iwai, Junji Kawakami   +6 more
doaj   +1 more source

Antisense Oligonucleotide-Mediated Removal of the Polyglutamine Repeat in Spinocerebellar Ataxia Type 3 Mice

Molecular Therapy: Nucleic Acids, 2017
Spinocerebellar ataxia type 3 (SCA3) is a currently incurable neurodegenerative disorder caused by a CAG triplet expansion in exon 10 of the ATXN3 gene.
Lodewijk J.A. Toonen, Frank Rigo, Haico van Attikum, Willeke M.C. van Roon-Mom   +3 more
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The Dynamics of Compound, Transcript, and Protein Effects After Treatment With 2OMePS Antisense Oligonucleotides in mdx Mice

Molecular Therapy: Nucleic Acids, 2014
Antisense-mediated exon skipping is currently in clinical development for Duchenne muscular dystrophy (DMD) to amend the consequences of the underlying genetic defect and restore dystrophin expression. Due to turnover of compound, transcript, and protein,
Ingrid E C Verhaart, Laura van Vliet-van den Dool, Jessica A Sipkens, Sjef J de Kimpe, Ingrid G M Kolfschoten, Judith C T van Deutekom, Lia Liefaard, Jim E Ridings, Steve R Hood, Annemieke Aartsma-Rus   +9 more
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Targeting several CAG expansion diseases by a single antisense oligonucleotide. [PDF]

PLoS ONE, 2011
To date there are 9 known diseases caused by an expanded polyglutamine repeat, with the most prevalent being Huntington's disease. Huntington's disease is a progressive autosomal dominant neurodegenerative disorder for which currently no therapy is ...
Melvin M Evers, Barry A Pepers, Judith C T van Deutekom, Susan A M Mulders, Johan T den Dunnen, Annemieke Aartsma-Rus, Gert-Jan B van Ommen, Willeke M C van Roon-Mom   +7 more
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Bolaamphiphile-based nanocomplex delivery of phosphorothioate gapmer antisense oligonucleotides as a treatment for Clostridium difficile

International Journal of Nanomedicine, 2016
John P Hegarty,1 Jacek Krzeminski,2 Arun K Sharma,2 Diana Guzman-Villanueva,3 Volkmar Weissig,3 David B Stewart Sr1 1Deparment of Surgery, Pennsylvania State University College of Medicine Hershey, PA, USA; 2Department of Pharmacology, Penn State ...
Hegarty JP, Krzeminski J, Sharma AK, Guzman-Villanueva D, Weissig V, Stewart DB Sr   +5 more
doaj  

Correction of Clcn1 alternative splicing reverses muscle fiber type transition in mice with myotonic dystrophy

Nature Communications, 2023
In a double homozygous mouse model of myotonic dystrophy type 1, Hu et al. use antisense oligonucleotide correction of myotonia to induce a therapeutic shift from an overabundance of oxidative muscle fibers to mechanically stronger glycolytic fibers.
Ningyan Hu, Eunjoo Kim, Layal Antoury, Thurman M. Wheeler   +3 more
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Nonviral delivery systems for antisense oligonucleotide therapeutics

Biomaterials Research, 2022
Antisense oligonucleotides (ASOs) are an important tool for the treatment of many genetic disorders. However, similar to other gene drugs, vectors are often required to protect them from degradation and clearance, and to accomplish their transport in ...
Si Huang, Xin-Yan Hao, Yong-Jiang Li, Jun‑Yong Wu, Da-Xiong Xiang, Shilin Luo   +5 more
doaj   +1 more source

When HERG-caused LQT2 encounters antisense oligonucleotide: is exon 6 skipping therapy plausible?

Frontiers in Pharmacology
Graphical AbstractThe unique in‐frame exon 6 of the HERG gene as a potential target for antisense oligonucleotide‐mediated exon skipping therapy.
Zequn Zheng, Zequn Zheng, Yongfei Song
doaj   +1 more source